Trial · NCT02678689
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT02678689, Phase 2, Completed, BioMarin Pharmaceutical; results first posted 2025-02-17
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease. Trials, record NCT02678689. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02678689
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
- NCT number
- NCT02678689
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 14 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
- Interventions
- BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device
- First posted
- 2016-02-10
- Last updated
- 2025-02-17
- Results first posted
- 2025-02-17
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Motor Language (ML) Scale: Rate of Decline in the 0 to 6-point ML Score. | Baseline to Last assessment (Week 169) | Rate of decline in 0 to 6-point ML score, \& primary analysis was based on up to 3-1 matching of Study 190-901 evaluable participants with Study 190-203 ITT participants. Rate of decline =(-1)x(48x7)x(Ending score - Starting score)/(Ending date - Starting date) A positive rate of decline means that subject declined, a negative rate of decline means that subject improved. The combined motor/gait and language (ML) score, as derived from Hamburg CLN2 rating scale, immediately preceding first infusion. The combined ML score is determined as sum of 0-3 point motor(M) \& language(L) subscales where 0 represents no function, \& 3 represents normal function, \& can range from 0(severely impaired) to 6(normal). Thus,high scores describe better function \& low scores describe poor function. The starting assessment is baseline ML assessment \& ending assessment is last ML score \>0. Note for Study 190-901, baseline ML assessment is defined as assessment of matching to Study 190-203 subj. |
| Primary | Probability of Unreversed 2-Point Decline in Motor-language (ML) Score or Score of 0 | Baseline to Last assessment (Week 169) | An unreversed 2-point decline is any decline of 2 points or more that had not reversed to a 1-point decline (or better) at last recorded observation. An unreversed score of 0 is a decline to 0 that had not increased to a score \>0 at last recorded observation ML score decline is measured by motor \& language domains on CLN2 rating scale. Combined motor/gait \&language (ML) score, as derived from Hamburg CLN2 rating scale, immediately preceding first infusion. Combined ML score is determined as sum of the 0-3 point motor(M)\&language(L) where 0 represents no function, \& 3 represents normal function, \& can range from 0 (severely impaired) to 6 (normal). Thus, high scores describe better function \& low scores describe poor function Model includes data up to week 169. Estimates from the model are presented for Wks 49, 97 \& 145 No.analyzed is no.of subj out of overall no.of participants analyzed who have not been censored/had unreversed 2-point decline/score of 0 at given time points. |
| Primary | Probability of Decline of Unreversed Motor-language (ML) Score of 0 | Baseline to Last assessment (Week 169) | The combined motor/gait and language (ML) score, as derived from the Hamburg CLN2 rating scale, immediately preceding the first infusion. The combined ML score is determined as the sum of the 0-3 point motor (M) and language (L) subscales where 0 represents no function, and 3 represents normal function, and can range from 0 (severely impaired) to 6 (normal). Thus, high scores describe better function and low scores describe poor function. Model includes data up to week 169. Estimates from the model are presented for Weeks 49, 97 and 145. No.analyzed is no.of subj out of overall no.of participants analyzed who have not been censored/had unreversed decline/score of 0 at given time points |
| Primary | Rate of Decline in Individual Motor Domains | Baseline to Last assessment (Week 169) | Rate of decline = (-1) x (48 x 7) x (Ending score - Starting score)/(Ending date - Starting date). A positive rate of decline means that the subject declined, a negative rate of decline means that the subject improved. The 0-3 point motor (M) subscales, as derived from the Hamburg CLN2 rating scale, where 0 represents no function, and 3 represents normal function. Thus, high scores describe better function, and low scores describe poor function. |
| Primary | Rate of Decline in Individual Language Domains | Baseline to Last assessment (Week 169) | Rate of decline = (-1) x (48 x 7) x (Ending score - Starting score)/(Ending date - Starting date). A positive rate of decline means that the subject declined, a negative rate of decline means that the subject improved. The 0-3 point language (L) subscales, as derived from the Hamburg CLN2 rating scale, where 0 represents no function, and 3 represents normal function. Thus, high scores describe better function and low scores describe poor function. Patients with baseline score of 0 are excluded. |
| Secondary | Probability of Decline of Disease Manifestation at Week 49 & 97 | Baseline to Last assessment (Week 169) | Disease manifestation is defined as post-baseline consecutive measurements of M,L,V/S scores\<3, measured atleast 22days apart. Combined motor-language-vision-seizure(MLVS)score as derived from Hamburg CLN2 rating scale, is determined as sum of 0-3 point motor(M)language(L)visio(V)\&seizure(S) subscales where 0 represents no function,\&3 represents normal function,\&can range from 0(severely impaired)to12(normal).Thus, high scores describe better function \& low scores describe poor function. 901 subj weighted according to no.of matches:weights for 3,2,\&1 study 901 subj matched to a given study 203 subj are 1/3,1/2, \&1 times N901/N203 respectively. N901 is no.of 901 subj matched to 203 subj(ie.18)\&N203 is no.203 subj who had matches (i.e.7).203 subj who had matches were assigned weight of 1. No.analyzed is no.of subj out of overall no.of participants analyzed who have not been censored/had decline of disease manifestation at given time points |
| Secondary | Probability of Decline of Disease Manifestation at Week 145 | Baseline to Week 145 | Disease manifestation is defined as post-baseline consecutive measurements of M,L,V/S scores\<3,measured atleast 22days apart Combined MLVS score,as derived from Hamburg CLN2 rating scale,is determined as sum of 0-3 point motor(M)language(L)vision(V)\&seizure(S) subscales where 0 represents no function,\&3 represents normal function,\&can range from 0(severely impaired) to 12(normal).Thus,high scores describe better function\&low scores describe poor function 901 subj weighted according to no.of matches:weights for 3,2,\&1 study 901 subj matched to given study 203 subj are 1/3,1/2, \&1 times N901/N203 respectively.N901 is no.of 901 subj matched to 203 subj(ie.18)\&N203 is no.203 subj who had matches(i.e.7).203 subj who had matches were assigned weight of 1 Model includes data upto wk169.Estimates from model are presented for Wk145 No.analyzed is no.of subj out of overall no.of participants analyzed who have not been censored/had decline of disease manifestation at given time point |
| Secondary | Change From Baseline in ML Scale Score | Baseline to Week 49, Week 97, Week 145, & Week 169 | The combined motor/gait and language (ML) score, as derived from the Hamburg CLN2 rating scale, immediately preceding the first infusion. The combined ML score is determined as the sum of the 0-3 point motor (M) and language (L) subscales where 0 represents no function, and 3 represents normal function, and can range from 0 (severely impaired) to 6 (normal). Thus, high scores describe better function and low scores describe poor function. Some participants did not have follow-up at all time points. |
| Secondary | Changes From Baseline in MLV Scale Score | Baseline to Week 49, Week 97, Week 145, & Week 169 | The combined motor-language-vision (MLV) score, as derived from the Hamburg CLN2 rating scale, is determined as the sum of the 0-3 point motor (M), language (L) \& vision (V) subscales where 0 represents no function, and 3 represents normal function, and can range from 0 (severely impaired) to 9 (normal). Thus, high scores describe better function and low scores describe poor function. Some participants did not have follow-up at all time points. |
| Secondary | Changes From Baseline in the 0-12 Point MLVS Motor, Language, Vision, and Seizure Subscales (MLVS) Score. | Baseline to Week 49, Week 97, Week 145, & Week 169 | The combined motor-language-vision-seizure (MLVS) score, as derived from the Hamburg CLN2 rating scale, is determined as the sum of the 0-3 point motor (M) and language (L) vision (V) and seizure (S) subscales where 0 represents no function, and 3 represents normal function, and can range from 0 (severely impaired) to 12 (normal). Thus, high scores describe better function and low scores describe poor function. Some participants did not have follow-up at all time points. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Motor Language (ML) Scale: Rate of Decline in the 0 to 6-point ML Score. | Baseline to Last assessment (Week 169) | Matched ITT BMN 190-203: Two subjects in 190-203 ITT population (N=14) were excluded from the 190-203 ITT analysis with matching (N=12) who did not match with any 190-901 subjects on the matching criteria. The matching criteria at baseline were: • Equal ML score • Age within 3 months • Genome: equal number of common alleles (c.622C→T, c.509.1G→C)~Matched 190-901 Natural history population: 29 subjects from DEM-CHILD database satisfying the criteria listed in the Arm/Group description. | change in score/48 wk | MEAN |
| PRIMARY | Probability of Unreversed 2-Point Decline in Motor-language (ML) Score or Score of 0 | Baseline to Last assessment (Week 169) | Matched ITT BMN 190-203: Two subjects in 190-203 ITT Population(N=14) were excluded from the 190-203 ITT analysis with matching (N=12) who did not match with any 190-901 subjects on the matching criteria. The matching criteria at baseline were: Equal ML score, age within 3 months and genome with equal number of common alleles(c.622C→T, c.509.1G→C).~Matched 190-901 Natural history population: 29 subjects from DEM-CHILD database satisfying the criteria listed in the Arm/Group description. | Probability of decline | NUMBER |
| PRIMARY | Probability of Decline of Unreversed Motor-language (ML) Score of 0 | Baseline to Last assessment (Week 169) | Matched ITT BMN 190-203: Two subjects in 190-203 ITT Population (N=14) were excluded from the 190-203 ITT analysis with matching (N=12) who did not match with any 190-901 subjects on the matching criteria. The matching criteria at baseline were: Equal ML score, age within 3 months and genome with equal number of common alleles (c.622C→T, c.509.1G→C).~Matched 190-901 Natural history population: 29 subjects from DEM-CHILD database satisfying the criteria listed in the Arm/Group description | Probability of decline | NUMBER |
| PRIMARY | Rate of Decline in Individual Motor Domains | Baseline to Last assessment (Week 169) | Matched ITT BMN 190-203: Two subjects in 190-203 ITT Population (N=14) were excluded from the 190-203 ITT analysis with matching (N=12) who did not match with any 190-901 subjects on the matching criteria. The matching criteria at baseline were: Equal ML score, age within 3 months and genome with equal number of common alleles (c.622C→T, c.509.1G→C).~Matched 190-901 Natural history population: 29 subjects from DEM-CHILD database satisfying the criteria listed in the Arm/Group description. | change in score/48 wk | MEAN |
| PRIMARY | Rate of Decline in Individual Language Domains | Baseline to Last assessment (Week 169) | Matched ITT BMN 190-203: Two subjects in 190-203 ITT Population (N=14) were excluded from the 190-203 ITT analysis with matching (N=12) who did not match with any 190-901 subjects on the matching criteria. The matching criteria at baseline were: Equal ML score, age within 3 months and genome with equal number of common alleles (c.622C→T, c.509.1G→C).~Matched 190-901 Natural history population: 29 subjects from DEM-CHILD database satisfying the criteria listed in the Arm/Group description. | change in score/48 wk | MEAN |
| SECONDARY | Probability of Decline of Disease Manifestation at Week 49 & 97 | Baseline to Last assessment (Week 169) | Matched ITT BMN190-203:2 subj in190-203 ITT Population(N=14)were excluded from 190-203 ITT analysis with matching(N=12)who did not match with any190-901 subj on matching criteria.Matching criteria at baseline were:Equal ML score,age\<3 months\&genome with equal no.of common alleles(c.622C→T,c.509.1G→C).~Matched190-901 Natural history population:29 subj from DEM-CHILD database satisfying criteria listed in Arm descp~Model includes data upto wk169.Estimates from model are presented for Wks 49\&97 | Probability of decline | NUMBER |
| SECONDARY | Probability of Decline of Disease Manifestation at Week 145 | Baseline to Week 145 | Matched ITT BMN190-203:2 subj in 190-203 ITT Pop(N=14) were excluded from 190-203 ITT analysis with matching(N=12) who did not match with any 190-901 subj on matching criteria. Matching criteria at baseline were:Equal ML score, age within 3 months \& genome with equal no. of common alleles(c.622C→T, c.509.1G→C).~Matched 190-901 Natural history pop:29 subj from DEM-CHILD database satisfying criteria listed in Arm/Group descp.~No.of subj analyzed for 190-901=0 \& hence this arm is not included | Probability of decline | NUMBER |
| SECONDARY | Change From Baseline in ML Scale Score | Baseline to Week 49, Week 97, Week 145, & Week 169 | 190-901 participants weighted according to no. of matches: weights for 3,2,\&1 study 190-901 participant matched to a given Study190-203 participant were1/3,1/2,\&1 times N901/N203, respectively. N901 was no. of 190-901 participants matched to 190-203 participants (ie,29) \&N203 was no. of 190-203 participants who had matches (ie,12).190-203 participants who had matches were assigned weight of 1.~Each participant in Study 190-901 had their scores imputed to time points using linear interpolation | score on a scale | MEAN |
| SECONDARY | Changes From Baseline in MLV Scale Score | Baseline to Week 49, Week 97, Week 145, & Week 169 | 190-901 participants weighted according to no. of matches: weights for 3,2,\&1 study 190-901 participant matched to a given Study190-203 participant were1/3,1/2,\&1 times N901/N203, respectively. N901 was no. of 190-901 participants matched to 190-203 participants (ie,29) \&N203 was no. of 190-203 participants who had matches (ie,12).190-203 participants who had matches were assigned weight of 1.~Each participant in Study 190-901 had their scores imputed to time points using linear interpolation | score on a scale | MEAN |
| SECONDARY | Changes From Baseline in the 0-12 Point MLVS Motor, Language, Vision, and Seizure Subscales (MLVS) Score. | Baseline to Week 49, Week 97, Week 145, & Week 169 | 190-901 participants weighted according to no. of matches: weights for 3,2,\&1 study 190-901 participant matched to a given Study190-203 participant were1/3,1/2,\&1 times N901/N203, respectively. N901 was no. of 190-901 participants matched to 190-203 participants (ie,29) \&N203 was no. of 190-203 participants who had matches (ie,12).190-203 participants who had matches were assigned weight of 1.~Each participant in Study 190-901 had their scores imputed to time points using linear interpolation | score on a scale | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group51
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Motor Language (ML) Scale: Rate of Decline in the 0 to 6-point ML Score. | BMN 190-203 | 0.15 | Standard Deviation 0.243 | change in score/48 wk | |
| Motor Language (ML) Scale: Rate of Decline in the 0 to 6-point ML Score. | Natural History Comparator: 190-901 Participants | 1.30 | Standard Deviation 0.857 | change in score/48 wk | |
| Probability of Unreversed 2-Point Decline in Motor-language (ML) Score or Score of 0 | BMN 190-203 | Probability of decline: Week 49 | 0.0 | 95% Confidence Interval 0.00 to 0.00 | Probability of decline |
| Probability of Unreversed 2-Point Decline in Motor-language (ML) Score or Score of 0 | Natural History Comparator: 190-901 Participants | Probability of decline: Week 49 | 0.141 | 95% Confidence Interval 0.05 to 0.35 | Probability of decline |
| Probability of Unreversed 2-Point Decline in Motor-language (ML) Score or Score of 0 | BMN 190-203 | Probability of decline: Week 97 | 0.083 | 95% Confidence Interval 0.01 to 0.46 | Probability of decline |
| Probability of Unreversed 2-Point Decline in Motor-language (ML) Score or Score of 0 | Natural History Comparator: 190-901 Participants | Probability of decline: Week 97 | 0.397 | 95% Confidence Interval 0.23 to 0.62 | Probability of decline |
| Probability of Unreversed 2-Point Decline in Motor-language (ML) Score or Score of 0 | BMN 190-203 | Probability of decline: Week 145 | 0.167 | 95% Confidence Interval 0.04 to 0.52 | Probability of decline |
| Probability of Unreversed 2-Point Decline in Motor-language (ML) Score or Score of 0 | Natural History Comparator: 190-901 Participants | Probability of decline: Week 145 | 0.774 | 95% Confidence Interval 0.59 to 0.92 | Probability of decline |
| Probability of Decline of Unreversed Motor-language (ML) Score of 0 | BMN 190-203 | Probability of decline: Week 49 | 0.0 | 95% Confidence Interval 0.00 to 0.00 | Probability of decline |
| Probability of Decline of Unreversed Motor-language (ML) Score of 0 | Natural History Comparator: 190-901 Participants | Probability of decline: Week 49 | 0.03 | 95% Confidence Interval 0.00 to 0.26 | Probability of decline |
1 to 10 of 51Source ClinicalTrials.gov
See all 51 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | BMN 190-203 | 14 | |
| Overall Study | STARTED | Natural History Comparator: 190-901 Participants | 29 | |
| Overall Study | COMPLETED | BMN 190-203 | 13 | |
| Overall Study | COMPLETED | Natural History Comparator: 190-901 Participants | 29 | |
| Overall Study | NOT COMPLETED | BMN 190-203 | 1 | |
| Overall Study | NOT COMPLETED | Natural History Comparator: 190-901 Participants | 0 |
Source ClinicalTrials.gov
Adverse events166
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Pyrexia | General disorders | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 12 | 14 | 62 |
| other | Upper respiratory tract infection | Infections and infestations | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 12 | 14 | 26 |
| other | Extensor plantar response | Nervous system disorders | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 7 | 14 | 7 |
| other | Gastroenteritis | Infections and infestations | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 7 | 14 | 10 |
| other | Generalised tonic-clonic seizure | Nervous system disorders | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 6 | 14 | 18 |
| other | Vomiting | Gastrointestinal disorders | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 5 | 14 | 7 |
| other | Contusion | Injury, poisoning and procedural complications | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 4 | 14 | 8 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 4 | 14 | 9 |
| other | Dysphagia | Gastrointestinal disorders | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 4 | 14 | 5 |
| other | Hypersensitivity | Immune system disorders | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 4 | 14 | 20 |
1 to 10 of 166Source ClinicalTrials.gov
See all 166 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 0 | 14 |
| Total, other adverse events | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 14 | 14 |
| Total, serious adverse events | BMN 190 Recombinant Human Tripeptidyl Peptidase-1 (rhTPP1) | 12 | 14 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02678689
GET https://conductscience.org/api/trials/v1/NCT02678689/results?page=2